Study on Mannan Binding Lectin (MBL) Substitution in MBL-Deficient Children With Chemotherapy-Induced Neutropenia

This study has been completed.
Sponsor:
Collaborator:
Landsteiner Foundation Blood Research (LSBR), Amsterdam
Information provided by:
Sanquin
ClinicalTrials.gov Identifier:
NCT00138736
First received: August 29, 2005
Last updated: August 29, 2007
Last verified: August 2007
  Purpose

The pharmacokinetics, and clinical and biological effects of MBL replacement therapy in MBL-deficient children during chemotherapy-induced neutropenia were studied.


Condition Intervention Phase
MBL-Deficient
Neutropenia
Drug: Mannan Binding Lectin (MBL)
Drug: Mannan Binding Lectin
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Pharmacokinetics Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Prevention
Official Title: Phase II Study on Mannan Binding Lectin (MBL) Substitution in MBL-Deficient Children With Chemotherapy-Induced Neutropenia

Resource links provided by NLM:


Further study details as provided by Sanquin:

Primary Outcome Measures:
  • pharmacokinetics of MBL [ Time Frame: until the patient's absolute neutrophil count (ANC) is above 500/uL blood. ]

Secondary Outcome Measures:
  • days of fever [ Time Frame: until the patient's absolute neutrophil count (ANC) is above 500/uL blood. ]
  • days of hospital admission [ Time Frame: until the patient's absolute neutrophil count (ANC) is above 500/uL blood. ]
  • use of antibiotics or antifungal medication [ Time Frame: until the patient's absolute neutrophil count (ANC) is above 500/uL blood. ]
  • number and type of infections [ Time Frame: until the patient's absolute neutrophil count (ANC) is above 500/uL blood. ]
  • MBL-dependent opsonizing capacity in vitro [ Time Frame: until the patient's absolute neutrophil count (ANC) is above 500/uL blood. ]
  • safety and incidence of side effects [ Time Frame: until the patient's absolute neutrophil count (ANC) is above 500/uL blood. ]

Enrollment: 12
Study Start Date: April 2004
Study Completion Date: October 2006
Arms Assigned Interventions
Experimental: A
MBL until the patient's absolute neutrophil count (ANC) is above 500/microL blood.
Drug: Mannan Binding Lectin (MBL) Drug: Mannan Binding Lectin

MBL dose at a twice weekly dose interval (3 or 4 days):

0.2 mg/kg, for a 3-day interval; 0.3 mg/kg, for a 4-day interval

Other Name: MBL SSI

Detailed Description:

Mannan Binding Lectin (MBL) is a member of the lectin pathway of the complement system and plays an important role in the innate immune system. MBL replacement in MBL-deficient children with chemotherapy-induced neutropenia represents a new approach to lower the risk of febrile episodes, of hospital admission, of prolonged use of intravenous antibiotics and of severe infections.

The aim of the Phase II study is to find evidence for the correct prediction of plasma levels of MBL necessary for clinical effects and biological efficacy, to confirm the dosage regimen needed to reach the required MBL plasma levels, and reconfirm the safety and lack of side-effects.

  Eligibility

Ages Eligible for Study:   up to 12 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Children ages 0 - 12 years, during chemotherapy, and expected to become neutropenic
  • MBL deficiency by genotype or phenotype (< 100 ng/ml)
  • Informed consent and assent of patient and/or legal representative

Exclusion Criteria:

  • Inability or unwillingness to comply with the protocol or likely inability to complete the study period
  • Known allergic reactions to MBL and other human plasma products
  • Participation in other investigational drug studies within the last month
  • Clinically relevant abnormalities in: serum immunoglobulins IgG, IgA, IgM; blood counts; complement factors measured by AP50, CH50; urine protein and cell counts; serum creatinine and liver enzymes, as routinely determined for regular patient care.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00138736

Locations
Netherlands
Academic Medical Centre
Amsterdam, Netherlands, 1105 AZ
Erasmus Medical Centre
Rotterdam, Netherlands, 3015 GD
Sponsors and Collaborators
Sanquin
Landsteiner Foundation Blood Research (LSBR), Amsterdam
Investigators
Principal Investigator: T W Kuijpers, MD, PdD Dept. of Paediatric Immunology, AMC, Amsterdam, The Netherlands
Principal Investigator: H N Caron Dept of Paediatric Oncology, AMC, Amsterdam, The Netherlands
  More Information

No publications provided

ClinicalTrials.gov Identifier: NCT00138736     History of Changes
Other Study ID Numbers: KB2003.02
Study First Received: August 29, 2005
Last Updated: August 29, 2007
Health Authority: Netherlands: The Central Committee on Research Involving Human Subjects (CCMO)

Keywords provided by Sanquin:
MBL-deficient
chemotherapy
neutropenia
chemotherapy-induced neutropenia

Additional relevant MeSH terms:
Neutropenia
Agranulocytosis
Leukopenia
Leukocyte Disorders
Hematologic Diseases

ClinicalTrials.gov processed this record on July 23, 2014